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Published in: Immunologic Research 1-3/2009

01-03-2009

Partially corrected X-linked severe combined immunodeficiency: long-term problems and treatment options

Authors: Suk See De Ravin, Harry L. Malech

Published in: Immunologic Research | Issue 1-3/2009

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Abstract

Rapid progress has been made from the identification of the molecular defects causing X-linked severe combined immune deficiency (X-SCID) to the development of cutting-edge therapeutic approaches such as hematopoietic stem cell transplant and gene therapy for XSCID. Successful treatment of XSCID has created a new population of patients, many of whom are now adolescents and young adults and are facing a variety of chronic problems secondary to partial correction of their underlying disease. This review focuses on the clinical challenges facing these patients (and their caregivers) and provides an overview of some of the treatment options available, including gene therapy.
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Metadata
Title
Partially corrected X-linked severe combined immunodeficiency: long-term problems and treatment options
Authors
Suk See De Ravin
Harry L. Malech
Publication date
01-03-2009
Publisher
Humana Press Inc
Published in
Immunologic Research / Issue 1-3/2009
Print ISSN: 0257-277X
Electronic ISSN: 1559-0755
DOI
https://doi.org/10.1007/s12026-008-8073-6

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